Rare dis­ease gene ther­a­py gains Prime sta­tus; FDA ap­proves first drug for in­flam­ma­to­ry arthri­tis nr-axS­pA

→ Pitts­burgh-based Krys­tal Biotech $KRYS has won the EMA’s Prime sta­tus for its gene ther­a­py to treat dy­s­troph­ic epi­der­mol­y­sis bul­losa. The com­pa­ny says reg­u­la­tors were won over by their on­go­ing GEM-1 Phase 1/2 study cou­pled with non-clin­i­cal da­ta.

→ The FDA on Thurs­day ap­proved the first treat­ment for nr-axS­pA, a type of arthri­tis that caus­es in­flam­ma­tion in the spine. The in­jec­tion, called Cimzia, has been de­vel­oped by UCB and was orig­i­nal­ly ap­proved in 2008 and is cur­rent­ly al­so sanc­tioned for use in Crohn’s dis­ease, rheuma­toid arthri­tis, ac­tive anky­los­ing spondyli­tis (AS) and plaque pso­ri­a­sis. The drug car­ries a boxed warn­ing for in­creased risk of se­ri­ous in­fec­tions lead­ing to hos­pi­tal­iza­tion or death, in­clud­ing tu­ber­cu­lo­sis, bac­te­r­i­al sep­sis, in­va­sive fun­gal in­fec­tions and oth­ers.

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